Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine - **Ticker Symbol**: IRON - **Public Trading Since**: End of 2022 - **Focus**: Development of therapies for blood disorders, particularly related to iron and heme metabolism [4][3] Key Programs and Developments Bitopertin - **Indication**: Erythropoietic protoporphyria (EPP) - **Regulatory Status**: Received a Commissioner's National Priority Voucher (CNPV), projecting potential approval by end of 2023 or early 2024 [2][5] - **Clinical Trials**: Completed a Phase 2 program with 100 participants; FDA aligned on an accelerated approval pathway using the toxic metabolite PPIX as a surrogate endpoint [5][6] - **Sales Force**: Planning to hire 24 sales representatives targeting 6,000 engaged patients identified through claims data [9][11] - **Market Size**: Estimated 14,000 diagnosed patients in the US, with a potential market size of $800 million to $1.4 billion [50] Competitive Landscape - **Existing Treatment**: Scenesse, which requires surgical implantation every two months, limiting its accessibility to about 3% of patients [12][14] - **Pricing Comparison**: Scenesse costs approximately $300,000 annually, while another treatment, Givlaari, is priced at $575,000 [18] Apollo Study - **Confirmatory Trial**: Enrolling 150 patients across the US, Europe, Canada, and Australia, expected to complete enrollment in about one year [21] - **Endpoints**: Co-primary endpoints include PPIX reduction and a measure of time in light at the end of the six-month trial [21] Other Assets 0974 - **Mechanism**: Monoclonal antibody targeting hemojuvelin to release iron for red blood cell production, primarily for myelofibrosis [22][23] - **Phase 1b Results**: Showed response rates of 50% in non-transfused patients, 80% in low transfusion burden patients, and 40% in high transfusion burden patients [26] - **Next Steps**: Interim results expected at ASH; aiming for a pivotal trial post-data analysis [37] Polycythemia Vera Program - **Goal**: Achieve iron restriction with a patient-friendly administration schedule [43] - **Phase 2 Study**: Aiming for a target hematocrit of 45% without phlebotomy, with a benchmark set against Rusfertide's 75% achievement [44][46] Financial Overview - **Cash Position**: Approximately $826 million post-stock offering, providing a runway until early 2029 without revenue from bitopertin [49] - **Investment Outlook**: Strong financial backing supports ongoing clinical trials and potential market entry [49] Additional Considerations - **Patient Engagement**: Focus on engaging the 6,000 identified patients initially, with broader outreach planned for the remaining diagnosed population [11] - **Regulatory Strategy**: Discussions with the FDA regarding the use of existing drug batches for potential early launch [6][8] This summary encapsulates the critical aspects of Disc Medicine's current status, focusing on their lead program, competitive landscape, financial health, and future plans.

Financial Data and Key Metrics Changes - The company is preparing to file its New Drug Application (NDA) for bitopertin in October, with a potential PDUFA date between June and October of the following year, marking a significant milestone for the company [4][5] - The company reported a P-value of less than 0.001 for the reduction in protoporphyrin-9 (PP9), indicating a clinically meaningful reduction in patients with erythropoietic protoporphyria [9][10] Business Line Data and Key Metrics Changes - The lead program, bitopertin, is in the accelerated approval pathway with the FDA, focusing on rare diseases related to red blood cell biology [4][5] - The second program is expected to present data on anemia of myelofibrosis and anemia of chronic kidney disease at upcoming conferences [5][6] Market Data and Key Metrics Changes - The company identified approximately 14,000 diagnosed patients in the U.S. for the target disease, with enrollment in trials not being a problem due to strong patient advocacy [18][23] - The company plans to leverage claims data to identify and validate physicians treating these patients, enhancing their market access strategy [23][24] Company Strategy and Development Direction - The company is focused on increasing disease awareness and ensuring that patients and physicians are informed about available therapies, as historically there have been no options for these patients [22][23] - The company aims to build a self-sustaining entity driven by revenues from bitopertin, with plans to explore additional indications within their iron modulation programs [60][61] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for bitopertin, citing positive interactions with the FDA regarding the use of PP9 as a surrogate endpoint [10][11] - The management highlighted the importance of continuous engagement with healthcare providers to ensure that patients receive appropriate care and treatment options [26][30] Other Important Information - The company has a strong financial position with $650 million on the balance sheet, which is expected to fund operations into 2028 [60] - The company has orphan protection for bitopertin, providing seven years of exclusivity in the U.S. plus potential pediatric extensions [36] Q&A Session Summary Question: Can you recap the interactions with the FDA regarding the accelerated approval path for bitopertin? - Management detailed a series of meetings with the FDA that led to the decision to pursue an accelerated approval pathway, emphasizing the strong rationale for using PP9 as a surrogate endpoint [10][11] Question: What are the key differences between the Apollo and Aurora trials? - Management explained that the Apollo trial will focus on measuring the time patients can spend in light, which is a clinically meaningful endpoint, alongside the reduction in PP9 levels [18][19] Question: How does the company plan to address the patient population for bitopertin? - Management discussed the importance of identifying engaged patients who regularly seek healthcare, estimating about 6,000 such patients, while also planning to reach out to the remaining diagnosed but less engaged patients [26][27] Question: What is the expected market opportunity for the company's anemia treatments? - Management indicated that there is a significant unmet need for anemia treatments in myelofibrosis, with an estimated market of about 22,000 patients, and potential pricing similar to existing therapies [42][44] Question: How does the company view its capital position and future financing needs? - Management expressed confidence in their capital position, indicating that they are well-funded to support ongoing trials and commercial preparations without immediate need for additional equity financing [60][61]

Financial Data and Key Metrics Changes - The company is preparing to file its New Drug Application (NDA) for bitopertin in October, with a potential PDUFA date between June and October of the following year, marking a significant milestone for the company [4][5][11] - The company reported a strong reduction in protoporphyrin-9 (PP9) levels with a P-value of less than 0.001, indicating clinically meaningful results from its phase 2 studies [9][10] Business Line Data and Key Metrics Changes - The lead program, bitopertin, is focused on treating erythropoietic protoporphyria, with plans for an accelerated approval pathway [4][5] - The second program is expected to present data on anemia of myelofibrosis and anemia of chronic kidney disease at upcoming conferences [5][6] Market Data and Key Metrics Changes - The company identified approximately 14,000 diagnosed patients in the U.S. for its lead indication, with expectations for rapid enrollment in clinical trials [19] - The market for bitopertin is expected to be significant, with pricing comparisons to existing therapies like Scenesse, which is priced at $300,000 to $350,000 per year [36] Company Strategy and Development Direction - The company is focused on increasing disease awareness and ensuring that healthcare providers are informed about available therapies for rare diseases [23][24] - The company plans to leverage claims data to identify and engage with physicians who treat diagnosed patients, enhancing its commercial strategy [24][25] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for bitopertin, citing positive interactions with the FDA regarding the use of PP9 as a surrogate endpoint [11][12] - The company is well-funded with $650 million on its balance sheet, providing a runway into 2028 to support ongoing trials and commercial preparations [62] Other Important Information - The company has orphan drug designation for bitopertin, providing seven years of market exclusivity in the U.S. [37] - The company is also exploring additional indications for its iron modulation programs, indicating a broad pipeline strategy [62] Q&A Session Summary Question: Can you recap the interactions with the FDA regarding the accelerated approval path for bitopertin? - Management detailed a series of meetings with the FDA that confirmed the rationale for an accelerated approval pathway based on phase 2 data and the use of PP9 as a surrogate endpoint [11][12] Question: What are the key differences between the Apollo and Aurora trials? - The Apollo trial will focus on measuring the time patients can spend in light as a co-primary endpoint, alongside the reduction of PP9 levels [19][20] Question: How does the company plan to address the patient population for bitopertin? - The company plans to segment the patient population based on engagement with healthcare providers, identifying approximately 6,000 engaged patients and 8,000 less engaged patients [27][28] Question: What is the expected pricing strategy for bitopertin? - The company anticipates pricing in line with existing therapies for rare diseases, potentially around $300,000 to $350,000 per year [36] Question: How does the company view the competitive landscape for its anemia treatments? - Management highlighted the significant unmet need in treating anemia in myelofibrosis, positioning their drug as the only one currently in development for this indication [43][44]